ITCC-101/APAL2020K, A Phase 1 trial of menin-inhibitor ziftomenib in combination with chemotherapy for children with relapsed/refractory KMT2A-r/NUP98-r/NPM1-m acute leukemia
ITCC-101/APAL2020K_Ziftomenib
What is the goal of the study?
Ziftomenib is an orally bioavailable, selective, and potent inhibitor of the binding between histone-lysine N-methyltransferase 2A (KMT2A) and menin. Nucleophosmin 1 gene (NPM1) alterations and nucleoporin (NUP98) gene alterations have been shown to rely on similar molecular pathways and respond to pharmacologic inhibition of the menin-KMT2A interaction. Ziftomenib has shown promising clinical efficacy in adult patients with relapsed/refractory KMT2A-rearranged (r) acute leukemias,as well as NUP98-r and NPM1-mutant (m) acute myeloid leukemia. Responses in children have emerged in early phase studies with revumenib, another menin inhibitor, but this drug has a different safety profile (see section 1.4). Especially, KMT2A-r and NUP98-r leukemias in children are considered high-risk and have a dismal outcome at relapse; hence, there is a clear medical need for improvement. Since differentiation syndrome is a known side effect of menin inhibition and is potentially fatal, this pediatric study will combine ziftomenib with standard-of-care chemotherapy in an attempt to mitigate this toxicity.
Who can participate in the study?
Please contact the study team listed below to learn more.